Mechanism explained- CRISPR Drug for Sickle Cell Anemia (Casgevy)

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I've heard a lot of people talking about this drug for sickle cell anemia but had a super hard time finding out the actual mechanism of how it works. So here is a high-level overview of everything you need to know to understand the basics of the first-ever CRISPR-based drug, enjoy!

Опубликовано:

27 май 2024

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Комментарии : 27

@sahwahhammad5219 Месяц назад

The explanation was very clear and easy to digest for both medical and those outside medical field. Regarding Sickle cell disease, it is best described as a missense mutation rather than SNPs because although it is a single nucleotide change it exists in less than 1% of population and it is associated with disease while SNPs are mostly non disease causing and exists in more than 1% of population.

@TheLabMouse Месяц назад

Thanks for the clarification! I had actually been taught about SNPs via sickle cell specifically. Likely since it is disease causing so it's easier to teach and resonates more. But you are right, nowadays I mainly hear the term SNPs thrown around to describe benign and nocoding regions.

@RockerProf 8 дней назад

Agreed! MTHFR can have SNPs. They naturally occur in about 40% of the population, and while not resulting in optimal proteins, are not technically a disease state. The sickle cell problem is caused by an infrequent missense mutation, one letter, which leads to a wrong amino acid, and being autosomal recessive, results in a disease state. A subtle but important distinction.

@isabelbrickner136 3 месяца назад

Didn’t even know about this happening, so I’m glad you made a video

@horacefields736 2 месяца назад

Very good explanation. I did need to pause the graphics, because there's a lot of information in them, but it's fascinating to see the mechanism.

@user-oh8hu8sm8z 3 месяца назад

Very interesting and I’d love to learn more.

@patrickdaoust01 15 дней назад

concise and thorough explanation - great job!

@kimquigleybrown 3 месяца назад

Brilliantly explained. Thank you for the education. Great job

@piyananboonprasirt110 2 месяца назад

Very clear explanation. Thank you!!

@shortfutiball7023 21 день назад

you've done an incredible job of explaning tyy

@ilovegod0106 3 месяца назад

Well explained. Thank you

@errolcampbell6985 Месяц назад

On my 58th year with sickle cell anemia...... they have done so much studies about it since I was a child..... I won't even think about any new treatment.....

@user-qn9xx2cv5t Месяц назад

What happens to the original defect beta-globin gene? Does removing BCL11a alone stop the expression of the beta-globin gene? If not, it seems there may still be issues with blood clotting.

@laurasolano1654 2 месяца назад

Thank you!! Appreciate it! You are the best. Big huge thank you!

@liyakhusnullina4310 2 месяца назад

Thank you so much!!

@samnoah9451 29 дней назад

We have already 2 million and 4 hundred thousand stems cells and we need 6 hundreds thousands more to make the stem cell transplantation well you pls pray for us to be free from sickell

@hadeerdodo823 Месяц назад

You are the best, thank you

@steveholmes1736 2 месяца назад

Well done

@PAC9322 2 месяца назад

Thanks for sharing...🥂

@andreazappacosta2906 27 дней назад

Hi, I was wondering if fetal Hb has an higher affinity to oxygen, wouldn’t this cause problems in oxygen exchange from capillaries to tissues? It would be like having Hb constantly at R state, which can get saturated very fast, but would also be very slow at giving away oxygen. I’m sorry if what I said makes no sense to you, I’m still a student😅

@marcxu6173 Месяц назад

People with sickle cell trait has mild symptoms, but with sickle cells disease, they need this medication.

@Elinondebra 6 дней назад

Meeting GOSTO HEB has really been one of the best day of my life after years of suffering I have been finally cured from cold sore thanks doc. I will always tell the world about your YT profile ❤❤🍀🍀❤❤❤❤❤ . . . . .